假肥大型肌营养不良症（DMD）是最常见的遗传性肌肉病，表现为进行性四肢近端骨骼肌萎缩无力，12岁不能行走，20岁左右死于呼吸衰竭。干细胞移植可使DMD模型鼠产生新的表达目的蛋白的肌细胞和运动功能改善，但新生肌细胞的神经支配怎样？神经肌肉接头有无功能？均不清楚。据以前的研究，我们提出"神经肌肉再生单位"假设，即干细胞移植后不仅有肌肉的再生，还有所支配的神经和血管再生。为证实"神经肌肉再生单位"假设，本研究拟分离DMD模型鼠的脂源性干细胞，基因修饰后经静脉移植，动态观察DMD模型鼠生命期限、运动功能、抗肌萎缩蛋白、病理、肌肉电生理变化；同时着重观察细胞移植后，是否可以形成供体来源的神经肌肉接头的突触前膜、后膜及新生血管，验证"神经肌肉再生单位"的存在，为干细胞移植治疗DMD将来应用于临床提供更有力的证据支持。这对于丰富干细胞移植后再生医学理论、阐明新生肌肉与与神经和血管再生的关系有重要意义。

Duchenne muscular dystrophy (DMD) is the most common genetic muscle disease. It is caused by a lack of functional dystrophin, a large membrane-associated protein encoded by DMD gene on X chromosome. This disease is characterized by progressive skeletal and cardiac muscle weakness. Afflicted individuals often die in their thirties, usually from respiratory and cardiac failure. In DMD mice model, muscle regeneration with functional dystrophin and amelioration of motor function were observed after stem cell transplantation. However, whether or not the regenerative muscle were under control with nerve? Does these new muscle presented neuromusclar junction? These question are still unclear.. We hypothesis that regenerative pattern of muscle after stem cell transplantation which we named neuromusclar regenerative nuit. In this study, to.confirm our hypothesis, we will isolate ADSCs of DMD mice model; correct its gene defect on dystrophin gene; and then inject in DMD model. We will evaluate the life span of mice model; expression of dystrophin; moter function; pathological and electrophysiological change on muscle. Furthermore, we will invertigate the neuromuscle junction; nerve timinal and change on blood supply of muscle. ALL these evidence are usefull to support our hypothesis.This study will certify that stem cell transplantation is safty and usefull to DMD therapy.